Software for Group-Sequential Trials

分组序贯试验软件

• 批准号: 7012716
• 负责人: Edward Lakatos
• 金额: $ 38.9万
• 依托单位: BIOSTATHAVEN, INC.
• 依托单位国家: 美国
• 财政年份: 2003
• 资助国家: 美国
• 起止时间: 2003-02-07 至 2008-01-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7012716
• 关键词: clinical trials; computer assisted medical decision making; computer assisted patient care; computer data analysis; computer graphics /printing; computer human interaction; computer program /software; computer system design /evaluation; drug screening /evaluation; ethics; experimental designs; human therapy evaluation; information display; mathematical model; patient safety /medical error; statistics /biometry

DESCRIPTION (provided by applicant): The term "group-sequential" is used to describe clinical trials in which statistical analyses will be conducted at various points over the course of the trial. If it can be shown at an interim analysis that the treatment is effective, there may be an ethical imperative to terminate the trial. Both the NIH and FDA mandate this type of trial for certain studies, including many in the field of heart disease, cancer, and AIDS. The goal of this project is to develop a comprehensive statistical software package for the design and analysis of group-sequential trials. It will provide modules for the design stage and the interim analysis stage. During the design stage, the user will be able to choose from a wide variety of stopping rules, and to calculate sample size and power. During the interim analysis stage, the user will be able to plot the current level of evidence in relation to the pre-specified boundary, as well as calculate conditional power. It will differ from what is currently available in several respects. First, it will use realistic survival models to accurately determine sample size and power. Other software force the user to assume that hazard rates are constant for the duration of the trial. In contrast, this program will allow the hazard rates and treatment effect to vary over time. In addition, it will allow researchers to account for such factors as non-compliance, and loss to follow-up and competing risks, etc. This will substantially improve estimates of sample size and power. It will also yield better estimates of the amount of information likely to be available at each of the interim analyses, which is crucial to be able to plan the timing of the DMC meetings and total study duration. It will provide tables and graphs for optimizing designs.

描述（由申请人提供）： 术语“组序”用于描述临床试验，其中在试验过程中将在各个方面进行统计分析。如果可以在临时分析中表明治疗是有效的，则可能有终止试验的道德要求。 NIH和FDA都要求进行某些研究，包括心脏病，癌症和艾滋病领域的许多试验。 该项目的目的是开发一个综合的统计软件包，用于设计和分析小组序列试验。它将为设计阶段和临时分析阶段提供模块。在设计阶段，用户将能够从各种停止规则中进行选择，并计算样本量和功率。在中期分析阶段，用户将能够绘制与预指定边界有关的当前证据水平，并计算有条件的功率。 它将与当前在几个方面可用的区别不同。首先，它将使用现实的生存模型来准确确定样本量和功率。其他软件迫使用户假设在试验期间危险率是恒定的。相比之下，该计划将允许随着时间的推移的危害率和治疗效果变化。此外，这将使研究人员能够说明诸如不合规之类的因素，以及在后续风险和竞争风险方面的损失等。这将大大改善样本量和权力的估计。它还将对每个临时分析中可能可用的信息量进行更好的估计，这对于能够计划DMC会议的时间和总研究持续时间至关重要。它将提供用于优化设计的表和图形。